RNA Therapeutics

ProQR is dedicated to discovering and developing RNA therapeutics for severe genetic rare diseases. Their QR-010 molecule is designed to address the most common mutation which causes cystic fibrosis. It has now demonstrated safety and clinical activity in two early clinical trials. This program integrated a creative approach to preclinical data, delivery, and clinical trial design to quickly advance this potential RNA therapeutic for a significant orphan disease population. Using a similar creative approach, ProQR have developed their second molecule, QR-110 for individuals with Leber’s congential amaurosis Type 10 into a now on-going clinical trial. Beyond that, have a pipeline of RNA therapeutics.

In the last several years, the RNA-based therapeutic development field has made good on the promise of decades of preclinical research. There are some outstanding and creative clinical trials using RNA-based therapeutics and strong momentum to bring these RNA-based therapeutics to patients.

For the detailed interview, check out the download centre online.

“I will share the challenges and choices made in bringing these molecules to development using QR-010 as the key example as it is farthest along.”
– Noreen Roth Henig

Hear more from Dr. Henig at RNA Therapeutics this February 2018, as she presents ‘RNA Therapeutics for Patients with Cystic Fibrosis: QR-010 and Beyond’

For those looking to attend there is currently a £400 early-bird saving, ending October 31st

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